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PRA’s Center for Rare Diseases understands that clinical trials are both a source of hope and care for people affected by a rare disease. The impact of COVID-19 on clinical trials is especially difficult for rare disease patient communities.

PRA Insights Team

April 23, 2020 – 5 min. read

Our deep connections to rare disease patient advocacy communities quickly enabled us to learn that many patient organizations wanted more information about the effect of COVID-19 on clinical trials. These communities felt more communication and transparency was needed. To respond to this need, the Center hosted two interactive webinars for rare disease patient organizations on Wednesday, April 1.

Advocates from more than 40 different rare disease areas, as well as from key global organizations, registered for the webinars. Attendees also completed a survey to let us know what key issues they currently face. Below, read some takeaways provided by rare disease patient organizations during our webinars and what they mean for rare disease trial sponsors.

We found three key takeaways from our webinars.

Impact on organizations

The most important challenges that organizations reported in the wake of the COVID-19 crisis were research slowdowns and organization/fundraising challenges.

Rare disease patient organizations are coping with a broad range of other organizational challenges, including reduced funding, canceled fundraising events, sharp increases in requests for information and support from the patients they serve, and navigating the shift to remote work.

Impact on clinical research

Participants voiced numerous concerns about COVID-19’s impact on rare disease clinical research. For most organizations, their greatest concerns about trials were general delays, abandonment, and delayed access to new experimental medicines.

A majority of the organizations also requested information about remote care/virtual trial practices that provide options for keeping clinical trials moving during a crisis.

Only 29% of those who registered reported being satisfied or very satisfied with the communication their organization received to date on COVID-19’s impact on clinical trials.

Impact on patient needs

Concerns about how patients’ unique care needs should be managed throughout the crisis were reported, ranging from access to care to confusion about medication.

Organizations reported that they would like to see sponsors of rare disease trials collaborate with them throughout the COVID-19 crisis. They suggested sponsors provide them with resources for their communities and regular status updates and involve them in evaluating the impact of COVID-19 on the patient community.

What does this mean for rare disease trial sponsors?

The Center for Rare Diseases believes effective collaboration with patient organizations is more important now than ever before, especially given the impact of the COVID-19 crisis on clinical programs.

For sponsors with ongoing clinical trials that are paused, collaboration with patient organizations can support the most efficient return to study activities beyond the crisis. Clinical trials are a vital source of care and hope for many rare communities, so sponsors can expect patient organizations to question what options exist to remotely continue some aspects of trials.

Sponsors who explore remote and virtual care models for current or upcoming trials will likely receive support from patient organizations. For sponsors who face lower levels of capitalization, or sponsors who are reassessing their early development pipelines, collaborating with patient organizations to assess the feasibility of clinical programs will be advantageous.

We can help you develop effective collaborative strategies with key patient organizations.

Sponsors are facing unprecedented challenges and adjusting to a constantly evolving pandemic situation.

As we navigate unprecedented challenges, the Patient Advocacy Strategy team at PRA’s Center for Rare Diseases is here to support the development of innovative medicines for rare diseases and the patients whose lives they may transform.